Gamida Cell Presents Data from Two Key Development Programs at the 2017 ASH Annual Meeting

Gamida
Cell, a leading cellular and immune therapeutics company, today
announced final results from the phase I/II trial evaluating NiCord, a
product derived from cord blood stem cells, as a stand-alone graft to
treat patients with high-risk hematologic malignancies. The study met
its primary endpoint, demonstrating rapid neutrophil engraftment with
manageable side effects. The company also presented preclinical data for
the advancement of natural killer cells (NK cells) as an
immunotherapeutic modality for patients with cancer. Both studies were
presented today at the annual meeting of the American Society of
Hematology (ASH) in Atlanta, GA.

“We are enthusiastic about the data presented today at ASH, including
the final results of our NiCord phase I/II study, which support the
basis for our global phase III trial of NiCord versus standard
unmanipulated cord blood transplantation (UCBT), currently enrolling
patients with hematologic malignancies,” said Julian Adams, Ph.D.,
chairman and chief executive officer of Gamida Cell. “We look forward to
further evaluating the potential of NiCord to serve as the graft of
choice for the thousands of patients with no matched donor in need of a
transplant every year, as well as continuing to progress our other
pipeline programs.”

Final Data from Phase I/II Trial of NiCord

The multicenter phase I/II study evaluated the safety and efficacy of
NiCord as a stand-alone graft in 36 patients with high-risk hematologic
malignancies, with a primary endpoint of time to neutrophil engraftment
following transplantation. Despite varying blood cancer diagnoses and
preparative conditioning regimens across patients across centers,
improved results were seen in the majority of study participants treated
with NiCord.

Final results of the study include the following:

Participants transplanted with NiCord had rapid and durable
engraftment of neutrophils and platelets, as well as prompt immune
reconstitution:

Median time to neutrophil engraftment was 11 days (95% CI: 9-13
days);

Median time to platelet engraftment was 34 days (95% CI: 32-42
days).

Results from the study participants were compared to a database of
matched patients from the Center for International Blood and Marrow
Transplant Research (CIBMTR). According to the CIBMTR data, patients
who received UCBT had a median time to neutrophil engraftment of 21
days and a median time to platelet engraftment of 46 days.

NiCord demonstrated an acceptable safety profile, with moderate/severe
chronic graft vs. host disease (cGvHD) in 9.8% of patients at one year
following transplantation. By day 100, 20.2% of participants experienced
grade 2-3 bacterial or grade 3 fungal infections.

“Historically, transplantation with cord blood has been limited due to
slow engraftment time in patients. We are looking to address this gap,
and this study demonstrated rapid and sustained engraftment in study
participants by utilizing technology to expand the number of stem cord
blood cells in a culture,” said Mitchell Horwitz, M.D., principal
investigator, co-study chair and professor of medicine at the Duke
Cancer Institute. “These phase I/II data demonstrate the potential to
make stem cell transplants accessible to a greater number of patients
who do not have a matched donor.”

Preclinical Data from NAM-NK Cell Program

Proof-of-concept data on the application of the company’s proprietary
NAM technology to healthy donor natural killer cells (NK cells) as a
potential immunotherapeutic approach to treating cancer were highlighted
in an oral presentation.

“The use of NK cells as a modality for immunotherapy has been limited by
impairedfunctionality of adoptively transferred NKcells
in patients,” said Ronit Simantov, M.D., chief medical officer at Gamida
Cell. “We are encouraged by the study results, which demonstrated
persistence and proliferation of NAM-NK cells in pre-clinical in vivo
models and describe a reliable, scalable culture model for the expansion
of functional donor NK cells aimed at clinical use.”

The analysis, which combines data from multiple preclinical studies,
validates the approach and is the basis for an investigator-sponsored,
phase I clinical trial of NAM-NK Cells in patients with
relapsed/refractory multiple myeloma or CD20-positive non-Hodgkin
lymphoma.

About NiCord

NiCord, the company’s lead clinical program, is under development as a
universal bone marrow transplant solution for patients with high-risk
hematologic malignancies. NiCord has demonstrated improved efficacy over
unmanipulated cord blood, including fewer bacterial and fungal
infections and a reduction in duration of hospital stays. NiCord has
been granted breakthrough status by the U.S. Food and Drug
Administration, making it the first bone marrow transplant alternative
to receive this designation. It has also received U.S. and EU orphan
drug designation. The ongoing phase III study is evaluating NiCord as a
curative treatment for patients with leukemia and lymphoma who have been
indicated for an allogeneic stem cell transplant. For more information
on NiCord clinical trials, please visit www.clinicaltrials.gov.

About NAM-NK Cells

Gamida Cell expanded the capabilities of its NAM technology to utilize
NK cells to create an immunotherapy to treat patients with refractor
blood cancers and solid tumors. Through expansion of highly functional
NK cells using NAM technology, NAM-NK Cells can be used to harness the
immune system to attack cancer. NAM-NK Cell is under phase I development
(NCT03019666) in patients with relapsed or refractory B-cell lymphoma
and multiple myeloma.

About Gamida Cell

Gamida Cell is a leader in cellular and immune therapeutics dedicated to
treating patients with cancer and rare genetic diseases. The company is
building a diverse pipeline based on its proprietary NAM technology
platform to deliver transformative medicines to patients in need of new
treatment options. To learn more about Gamida Cell, including current
clinical studies, please visit www.gamida-cell.com
and on Twitter,
LinkedIn
and Facebook.

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